KR20240035502A - 신독성 경감제 - Google Patents

신독성 경감제 Download PDF

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Publication number
KR20240035502A
KR20240035502A KR1020247003682A KR20247003682A KR20240035502A KR 20240035502 A KR20240035502 A KR 20240035502A KR 1020247003682 A KR1020247003682 A KR 1020247003682A KR 20247003682 A KR20247003682 A KR 20247003682A KR 20240035502 A KR20240035502 A KR 20240035502A
Authority
KR
South Korea
Prior art keywords
nephrotoxicity
antisense oligomer
sugar
pharmaceutical composition
antisense
Prior art date
Application number
KR1020247003682A
Other languages
English (en)
Korean (ko)
Inventor
다카시 호리우치
Original Assignee
니뽄 신야쿠 가부시키가이샤
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 니뽄 신야쿠 가부시키가이샤 filed Critical 니뽄 신야쿠 가부시키가이샤
Publication of KR20240035502A publication Critical patent/KR20240035502A/ko

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7016Disaccharides, e.g. lactose, lactulose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00General protective or antinoxious agents
    • A61P39/02Antidotes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
KR1020247003682A 2021-07-08 2022-07-08 신독성 경감제 KR20240035502A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JPJP-P-2021-113563 2021-07-08
JP2021113563 2021-07-08
PCT/JP2022/027104 WO2023282345A1 (ja) 2021-07-08 2022-07-08 腎毒性軽減剤

Publications (1)

Publication Number Publication Date
KR20240035502A true KR20240035502A (ko) 2024-03-15

Family

ID=84800761

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247003682A KR20240035502A (ko) 2021-07-08 2022-07-08 신독성 경감제

Country Status (8)

Country Link
EP (1) EP4368186A1 (pt)
JP (2) JPWO2023282345A1 (pt)
KR (1) KR20240035502A (pt)
AU (1) AU2022306820A1 (pt)
CA (1) CA3225454A1 (pt)
IL (1) IL310003A (pt)
TW (1) TW202337473A (pt)
WO (1) WO2023282345A1 (pt)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002036767A2 (en) 2000-11-02 2002-05-10 Inex Pharmaceuticals Corporation Therapeutic oligonucleotides of reduced toxicity
WO2010009038A2 (en) 2008-07-18 2010-01-21 Oncogenex Technologies Inc. Antisense formulation
JP2015091229A (ja) 2013-10-04 2015-05-14 国立大学法人神戸大学 福山型筋ジストロフィー治療用アンチセンス核酸

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EP0962463B1 (en) 1989-12-20 2002-07-10 Antivirals Inc. Uncharged morpholino-based polymers having phosphorus-containing chiral intersubunit linkages
JPWO2006038608A1 (ja) 2004-10-05 2008-05-15 日本新薬株式会社 オリゴ二本鎖rna及び医薬組成物
EP1811960A2 (en) * 2004-11-19 2007-08-01 Novosom AG Improvements in or relating to pharmaceutical compositions for local administration
WO2006129594A1 (ja) 2005-05-30 2006-12-07 Nippon Shinyaku Co., Ltd. 核酸含有複合体製剤の製造方法
ES2657400T3 (es) 2006-05-10 2018-03-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
CN1868456A (zh) * 2006-06-25 2006-11-29 沈阳药科大学 反义脱氧寡核苷酸isis23665脂质体制剂及制备方法
WO2009005793A2 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
CA2884340C (en) 2007-11-15 2017-07-25 Sarepta Therapeutics, Inc. Method of synthesis of morpholino oligomers
CA2930393C (en) 2007-12-04 2022-11-29 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CN103619356B (zh) 2011-05-05 2017-09-12 萨勒普塔医疗公司 肽寡核苷酸缀合物
CN102989007B (zh) * 2011-06-29 2015-12-16 湖州市中心医院 大规模制备注射用中期因子反义寡核苷酸纳米脂质体的方法
SG11201402392QA (en) 2011-11-18 2014-06-27 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
RU2619184C2 (ru) 2011-12-28 2017-05-12 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
RU2019110030A (ru) 2013-05-01 2019-05-06 Ионис Фармасьютикалз, Инк. Композиции и способы
CA2917161C (en) 2013-07-11 2023-12-12 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
US9856496B2 (en) 2013-12-12 2018-01-02 Life Technologies Corporation Membrane-penetrating peptides to enhance transfection and compositions and methods for using same
JP6482475B2 (ja) 2014-01-07 2019-03-13 レナセラピューティクス株式会社 アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド
WO2016187425A1 (en) 2015-05-19 2016-11-24 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US20180193471A1 (en) 2015-07-16 2018-07-12 Kyowa Hakko Kirin Co., Ltd. ß2GPI GENE EXPRESSION-SUPPRESSING NUCLEIC ACID CONJUGATE
SI3554554T1 (sl) 2016-12-19 2023-02-28 Sarepta Therapeutics, Inc. Oligomerni konjugati, ki preskakujejo ekson za mišično distrofijo
WO2018118599A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
HUE059905T2 (hu) 2016-12-19 2023-01-28 Sarepta Therapeutics Inc Exonátugró oligomerkonjugátumok izomdisztrófiára

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002036767A2 (en) 2000-11-02 2002-05-10 Inex Pharmaceuticals Corporation Therapeutic oligonucleotides of reduced toxicity
WO2010009038A2 (en) 2008-07-18 2010-01-21 Oncogenex Technologies Inc. Antisense formulation
JP2015091229A (ja) 2013-10-04 2015-05-14 国立大学法人神戸大学 福山型筋ジストロフィー治療用アンチセンス核酸

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Carver MP, Charleston JS, Shanks C, Zhang J, Mense M, Sharma AK, Kaur H, Sazani P. Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates. J Neuromuscul Dis. 2016 Aug 30;3(3):381-393. doi:10.3233/JND-160157.
Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ, van Ommen GJ, van Deutekom JC, Aartsma-Rus A. In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med. 2009 Mar;11(3):257-66. doi:10.1002/jgm.1288.
Sazani P, Ness KP, Weller DL, Poage D, Nelson K, Shrewsbury AS. Chemical and mechanistic toxicology evaluation of exon skipping phosphorodiamidate morpholino oligomers in mdx mice. Int J Toxicol. 2011 May;30(3):322-33. doi:10.1177/1091581811403504. Epub 2011 May 3.

Also Published As

Publication number Publication date
AU2022306820A1 (en) 2024-01-04
JP7466610B2 (ja) 2024-04-12
EP4368186A1 (en) 2024-05-15
TW202337473A (zh) 2023-10-01
JP2023130287A (ja) 2023-09-20
IL310003A (en) 2024-03-01
WO2023282345A1 (ja) 2023-01-12
CA3225454A1 (en) 2023-01-12
JPWO2023282345A1 (pt) 2023-01-12

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